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Compositions and methods for the treatment of myotonic dystrophy

A genome and repetitive sequence technology, applied in the field of compositions for the treatment of myotonic dystrophy, can solve the problems of unsuitable removal of trinucleotide repeat sequence expansion regions and the like

Pending Publication Date: 2019-10-15
GENETHON +1
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

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Problems solved by technology

However, the latter is considered by those skilled in the art to be unsuitable for excision of trinucleotide repeat expansions (see Richard cited above)

Method used

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  • Compositions and methods for the treatment of myotonic dystrophy
  • Compositions and methods for the treatment of myotonic dystrophy
  • Compositions and methods for the treatment of myotonic dystrophy

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Experimental program
Comparison scheme
Effect test

Embodiment

[0138] A table matching the SEQ ID NOs to the sgRNA numbers used in the experimental section and figures below is provided below.

[0139] sgRNA ID 1 4 7 8B 12 12B SEQ ID NO 1 20 3 4 21 6

[0140] Materials and methods

[0141] Plasmid construction

[0142] The plasmid encoding S. aureus Cas9 was derived from the plasmid pX601-AAV-CMV::NLS-SaCas9-NLS-3xHA-bGHpA;U6::BsaI-sgRNA(MLS42) [Ran et al., 2015]. The EFS promoter was PCR amplified with primers F-XhoI-MreI-EFS (MLS63) and R-XmaI-NruI-EFS (MLS64) and cloned into the promoterless pX601-AAV-::NLS-SaCas9- NLS-3xHA-bGHpA;U6::BsaI-sgRNA XhoI / AgeI sites to obtain pAAV-EFS::NLS-SaCas9-NLS-3xHA-bGHpA;U6::BsaI-sgRNA(MLS43).

[0143] The second sgRNA cassette (U6::BbsI-sgRNA) was cloned in tandem into the Acc65I site upstream of the first sgRNA cassette in plasmid MLS43 to obtain construct pAAV-EFS::NLS-SaCas9-NLS-3xHA-bGHpA; U6::BbsI-sgRNA; U6::BsaI-sgRNA (MLS47). Insert U6::BbsI-sgRNA was synt...

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Abstract

The present invention relates to compositions and methods for the treatment of myotonic dystrophy.

Description

technical field [0001] The present invention relates to compositions and methods for treating myotonic dystrophy. Background technique [0002] Nucleotide repeat expansions, especially trinucleotide repeat expansions, have been implicated in more than twenty neurological and developmental disorders. One approach to treating these diseases has been proposed to use highly specific nucleases to shorten repeat sequences to nonpathological lengths (see review by Richard GF, Trends Genet. 2015 Apr;31(4):177-186). [0003] Highly specific nucleases such as meganucleases, ZFNs, TALENs and CRISPR-Cas9 nucleases have been used in such strategies. However, those skilled in the art consider the latter to be unsuitable for excision of trinucleotide repeat expansions (see Richard cited above). In conclusion, TALENs are considered to be more promising tools for shortening trinucleotide repeats. [0004] Against this strong bias, the inventors here show that a CRISPR-Cas9 system can be i...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): C12N15/113C12N9/22C12N15/63C12N15/90A61P21/00C12N15/11
CPCC12N9/22C12N15/113C12N15/907C12N2310/10C12N2310/20C12N15/90C12N2750/14141A61P21/00A61P21/04A61P43/00A61K9/0019A61K35/12C12N15/86
Inventor 安娜·玛利亚·布吉贝洛米雷拉·洛斯克鲁达托
Owner GENETHON
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